Comparison of efficacy and tolerability of azathioprine, mycophenolate mofetil, and cyclophosphamide among patients with neuromyelitis optica spectrum disorder: A prospective cohort study

Comparison of efficacy and tolerability of azathioprine, mycophenolate mofetil, and cyclophosphamide among patients with neuromyelitis optica spectrum disorder: A prospective cohort study
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DOI:
10.1016/j.jns.2016.09.035
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发表时间:
2016-11-15
影响因子:
4.4
通讯作者:
Cui, Li-ying
Cui, Li-ying
中科院分区:
医学3区
文献类型:
--
作者:
Xu, Yan;Wang, Qian;Cui, Li-ying

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目的:比较硫唑嘌呤(AZA)、霉酚酸酯(MMF)和环磷酰胺(CTX)治疗视神经脊髓炎光谱障碍(NMOSD)的疗效和耐受性。方法:对接受AZA、MMF和CTX治疗的NMOSD患者(n=119、38和41)的复发、残疾和不良事件进行前瞻性队列分析。结果:服用氮卓酮(P<0.001)、霉酚酸酯(P<0.001)或环磷酰胺(P=0.001)的患者复发率显著降低。MMF与AZA相比,复发风险较低,但这种差异没有统计学意义(p=0.08)。AZA和MMF显著降低了扩展残疾状态量表(EDSS)的平均得分(AZA:P=0.02;MMF:P=0.01),而CTX则没有。与AZA相比,MMF因药物相关不良事件而中断治疗的风险显著降低(p=0.02),而CTX的风险与AZA相似(p=0.35)。结论:MMF是治疗NMOSD的良好一线药物,如果其副作用可耐受,AZA仍是一种有价值的一线药物,而CTX可作为不耐受AZA和MMF的患者的一种治疗方案。(C)2016爱思唯尔B.V.保留所有权利。
Aims: To compare the efficacy and tolerability of azathioprine (AZA), mycophenolate mofetil (MMF), and cyclophosphamide (CTX) in patients with neuromyelitis optica spectrum disorder (NMOSD).Methods: We performed a prospective cohort analysis of relapses, disability, and adverse events in NMOSD patients treated with AZA, MMF, or CTX (n = 119, 38, and 41, respectively). All the patients were co-treated with oral prednisone.Results: A significant reduction in relapse rate was found in patients taking AZA (p < 0.001), MMF (p < 0.001) or CTX (p = 0.01). MMF was associated with a lower risk of relapse than AZA, but this difference was not statistically significant (p = 0.08). AZA and MMF decreased the mean Expanded Disability Status Scale (EDSS) scores significantly (AZA: p = 0.02; MMF: p = 0.01), whereas CTX did not. Compared with AZA, MMF had a significantly lower risk of treatment discontinuation due to drug-related adverse events (p = 0.02), whereas CTX had a comparable risk (p = 0.35).Conclusions: MMF is a good first-line treatment option for NMOSD and AZA remains a valuable first-line drug if its side effects are tolerable while CTX can be a treatment option for patients who cannot tolerate AZA and MMF. (C) 2016 Elsevier B.V. All rights reserved.